Transposon Receives US FDA Fast Track Designation for TPN-101 for Progressive Supranuclear Palsy

Transposon Receives US FDA Fast Track Designation for TPN-101 for Progressive Supranuclear Palsy

May 21, 2024 / Portfolio News

Designation Supports Accelerated Drug Development for PSP
TPN-101 is the First PSP Treatment to Reduce NfL and IL-6 Levels,
Key Biomarkers of Neurodegeneration and Neuroinflammation in PSP


SAN DIEGO, California, May 21, 2024 – Transposon Therapeutics, a biotechnology company
developing a platform of novel, orally administered therapies for the treatment of
neurodegenerative and aging-related diseases, today announced that the U.S. Food and Drug
Administration (FDA) has granted Fast Track designation to TPN-101 for progressive
supranuclear palsy (PSP).


“Fast Track designation for TPN-101 is an important acknowledgement by the FDA of the
critical need to find an effective treatment for PSP, a rare and devastating neurological disorder
with no approved treatment options,” said Dennis Podlesak, Chairman and Chief Executive
Officer of Transposon. “We look forward to working collaboratively with the FDA to advance
the development of TPN-101 as rapidly as possible for the treatment of PSP and other
neurodegenerative diseases including ALS and Alzheimer’s disease.”


The designation of TPN-101 as a Fast Track product for PSP is supported by data from a Phase
2, randomized, double-blind, placebo-controlled study of TPN-101 in patients with PSP. In that
study, TPN-101 was the first treatment for PSP to reduce levels of neurofilament light chain
(NfL), a key biomarker of neurodegeneration in tauopathies such as PSP and Alzheimer’s
disease. TPN-101 also showed dose-related reductions in interleukin 6 (IL-6) cytokine levels, a
biomarker of neuroinflammation that is elevated in PSP and correlates with disease progression
and severity. Participants treated with TPN-101 for the entire 48-week trial duration showed a
stabilization of their clinical symptoms as measured by the PSP Rating Scale (PSPRS) between
weeks 24 and 48. Further information on the study can be accessed at ClinicalTrials.gov.


About Fast Track Designation
The FDA grants Fast Track designation to facilitate the development and expedite the review of
medicines to treat serious conditions and fill an unmet medical need. Fast Track status allows
for enhanced communication and collaboration between the FDA and drug developers,
potentially speeding up the delivery of life-saving treatments to patients.
About PSP
PSP is a rare neurodegenerative disorder that causes slowing of movement, loss of balance
leading to falls, impaired eye movements, and disturbances in cognition and behavior. The
disease typically affects people in their mid- to late-60s, and the mean survival for individuals

with PSP is 6 to 7 years. There are currently no treatments capable of delaying the progression
of the disease.


About Transposon
Transposon Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a
platform of novel therapies for the treatment of neurodegenerative and aging-related diseases,
including progressive supranuclear palsy, amyotrophic lateral sclerosis, frontotemporal
dementia, and Alzheimer’s disease. The company’s lead clinical compound, TPN-101, is first-inclass
to address LINE-1 reverse transcriptase for treating neurodegenerative and autoimmune
diseases. The company also has a discovery platform supporting a deep pipeline of novel
therapies to address additional indications.


About TPN-101
TPN-101 specifically inhibits the LINE-1 reverse transcriptase that promotes LINE-1 replication.
LINE-1 elements are a class of retrotransposable elements that in humans are uniquely capable
of replicating and moving to new locations within the genome. When this process becomes
dysregulated, LINE-1 reverse transcriptase drives overproduction of LINE-1 DNA, triggering
innate immune responses that contribute to neurodegenerative, autoimmune and aging-related
disease pathology.


Contact:
Rick Orr
Transposon Therapeutics, Inc.
(858) 535-4821
rorr@transposonrx.com

Orthofix Announces Agreement to Acquire Spinal Kinetics

March 15, 2018 / Portfolio News

Transaction to expand Orthofix’s Spine Fixation portfolio with innovative artificial disc designed to restore natural and physiologic motion to the spine.

LEWISVILLE, Texas–(BUSINESS WIRE)–Orthofix International N.V. (NASDAQ:OFIX), a global medical device company focused on musculoskeletal healing products and value-added services, today announced that it has entered into a definitive agreement to acquire Spinal Kinetics Inc., a privately held developer and manufacturer of artificial cervical and lumbar discs. Terms of the agreement include $45 million in cash closing consideration plus up to $60 million in contingent milestone payments related to U.S. Food and Drug Administration approval of the M6-C cervical disc and the achievement of trailing twelve-month sales targets of $30 million and $50 million.

“The Spinal Kinetics M6 artificial discs will further strengthen Orthofix’s product portfolio by filling a strategic gap in our Spine Fixation product line. This technology is a significant advancement in mimicking the natural motion of the spine, which we believe will be very beneficial to patients and well received by our surgeon customers,” said Orthofix President and Chief Executive Officer, Brad Mason. “This acquisition is very well aligned with our value creation strategy of accelerating topline growth by investing in faster growing market segments in our core businesses. In addition, we expect this news will energize our sales force and be attractive to potential new sales talent.”

Spinal Kinetics manufactures and distributes the M6-C cervical and M6-L lumbar artificial discs for patients suffering from degenerative disc disease (DDD) of the spine. These unique discs are designed to mimic the anatomic structure of a natural disc by incorporating an artificial visco-elastic nucleus and fiber annulus. This allows for six degrees of motion, similar to a natural disc.

“Artificial disc replacement is increasingly being indicated as the superior surgical solution to the traditional spinal fusion because it maintains normal motion of the spine and in many cases lessens the chance of future surgery. However, the designs of the first-generation artificial discs, much like total hip replacement, were based on the ball-and-socket concept which does not take into account the natural compression of the native disc,” said Dr. Richard D. Guyer, orthopedic spine surgeon and Chairman of the Texas Back Institute Research Foundation in Dallas and an investigator in the “Restore” U.S. clinical trial sponsored by Spinal Kinetics. “The M6 disc is designed out of materials to mimic the biomechanics of a normal disc including axial compression, flexion-extension, lateral bending, translation and axial rotation in order to provide patients with a more natural range of motion.”

The M6 artificial discs currently have CE Mark approval for distribution in the European Union and other international geographies. They are not available for commercial distribution in the U.S. Spinal Kinetics has submitted a PMA to the U.S. Food and Drug Administration in order to gain U.S. market approval for the M6-C cervical disc to treat single level cervical DDD. Internationally, there have been more than 54,000 implants of the M6-C and M6-L since the products were first launched in 2006.

“We look forward to becoming a part of the Orthofix team,” said Tom Afzal, President and CEO of Spinal Kinetics. “Joining forces gives us the opportunity to bring together Spinal Kinetics’ proven innovative technology with Orthofix’s regulatory, market development, distribution and commercial expertise as we work to broaden the availability of these devices and ultimately prepare for U.S. commercialization.”

Orthofix estimates the artificial disc market in 2017 to be over $325 million worldwide and $200 million in the U.S., with double-digit growth expected for many years. Also, Orthofix anticipates that the momentum created from the addition of the M6 disc to the Orthofix spine fixation portfolio will generate pull-through revenue of other Orthofix products and position the company for market share gains in the $5.4 billion U.S. spine hardware market.

The transaction is anticipated to close in the second quarter of 2018, subject to customary closing conditions. Orthofix expects the acquisition to not only add revenue in 2018, but also increase its organic revenue growth rate in 2019 and beyond. The company also expects the deal to be slightly accretive to the Company’s non-GAAP diluted earnings per share and adjusted EBITDA within 12 months of PMA approval in the U.S. and further accretive thereafter.

In connection with the transaction, Canaccord Genuity is acting as a financial advisor to Spinal Kinetics.

Orthofix Conference Call

Orthofix will conduct a conference call on Thursday, March 15 at 4:00 p.m. Central time (5:00 p.m. Eastern time). An overview of the transaction will be provided during the call. The investor presentation is viewable on Orthofix’s U.S. corporate home page or ir.orthofix.com. Interested parties may access the conference call by dialing (844) 809-1992 in the U.S. and (612) 979-9886 outside the U.S., and referencing the conference ID 7493218. A replay of the call will be available for two weeks by dialing (855) 859-2056 in the U.S. and (404) 537-3406 outside the U.S., and entering the conference ID 7493218.

About Orthofix

Orthofix International N.V. is a global medical device company focused on musculoskeletal healing products and value-added services. The Company’s mission is to improve patients’ lives by providing superior reconstruction and regenerative musculoskeletal solutions to physicians worldwide. Headquartered in Lewisville, Texas, the Company has four strategic business units: BioStim, Extremity Fixation, Spine Fixation, and Biologics. Orthofix products are widely distributed via the Company’s sales representatives and distributors. For more information, please visit www.orthofix.com.

About Spinal Kinetics Inc.

Founded in 2003, Spinal Kinetics is a privately held medical device company focused on partnering with spine surgeons to develop innovative and practical motion preservation systems for treating degenerative diseases of the spine. The M6-C cervical and M6-L lumbar artificial discs have rapidly established themselves among the leading artificial discs available due to the unique biomechanical properties that replicate the motion of a natural disc and the positive clinical outcomes for patients. The company is located in Sunnyvale, California. For more information about Spinal Kinetics or the M6 Artificial Disc, please visit spinalkinetics.com.

Forward Looking Statements

This communication contains certain forward-looking statements under the Private Securities Litigation Reform Act of 1995. These forward-looking statements, which may include, but are not limited to, statements concerning the estimates, projections, financial condition, results of operations and businesses of Orthofix and its subsidiaries, Spinal Kinetics and their respective companies’ product portfolios, are based on Orthofix management’s current expectations and estimates and involve risks and uncertainties that could cause actual results or outcomes to differ materially from those contemplated by the forward-looking statements.

The forward-looking statements in this release do not constitute guarantees or promises of future performance. Factors that could cause or contribute to such differences may include, but are not limited to risks, including the possibility that the deal might not close, difficulties commercializing Spinal Kinetics’ products and integrating their product lines into Orthofix’s business, inaccuracies in Orthofix’s estimates and projections of future product sales, including the current and future size of the worldwide and U.S. artificial disc market, FDA and regulatory approval risks, and other risks described in the “Risk Factors” section of our 2017 Annual Report on Form 10-K, as well as in other reports that we file in the future. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. The Company undertakes no obligation to update or revise the information contained in this press release.

Contacts

Orthofix International N.V.
Investor Relations
Mark Quick, 214-937-2924
markquick@orthofix.com
or
Media Relations
Denise Landry, 214-937-2529
deniselandry@orthofix.com

This exec tested his company’s medical imaging tech–and found he had cancer

December 1, 2017 / Portfolio News

“Technology can save your life” is a threadbare cliché. But for Rob Sandler, a 50-year-old senior marketing executive at medical imaging firm Exact Imaging, that overused phrase transformed overnight into a sacred truth. During a routine test of a high-resolution ultrasound machine developed by the company, it spotted something wrong: Sandler had aggressive prostate cancer.

Cancer of the prostate is the most common type of cancer in men. If detected early, it is extremely treatable. But if left undiagnosed, significant prostate cancer can invade other organs and tip the odds against the patient — five-year survival is then a dismal 29 per cent. The trouble is, prostate cancer can be difficult to detect early as doctors are essentially looking for a tiny lump on an organ the size of a walnut.

Exact Imaging has developed a high-resolution micro-ultrasound that helps doctors to detect possible prostate cancer and enables them to better target biopsies. It has so far sold 25 systems around the world at the price tag of $150,000 per machine and is also working on an artificial intelligence algorithm to further hone its detection abilities.

Chance discovery

As part of their product development, Exact Imaging’s engineers sometimes ask senior managers to act as model patients to test the micro-ultrasound scanner. One day this past summer, a tap on Sandler’s office window told him it was his turn to volunteer.

His eighth time being scanned, Sandler thought nothing of it as he made his way to the lab. But as the scan was being conducted, it was clear that this time something wasn’t right. There were unmistakable lesions on the high-resolution images, serious enough for them to be immediately dispatched to a uro-radiologist at Princess Margaret Cancer Centre in Toronto.

After further tests, the results were indisputable. Sandler had a very aggressive cancer that probably would have spread like wildfire in a few months if not treated immediately. “I wouldn’t be sitting here if it wasn’t for me volunteering for that random, inadvertent scan done by our micro-ultrasound,” says Sandler.

Slipping through the net

To appreciate Exact Imaging’s technology and Sandler’s near-brush with death, it is helpful to consider how convoluted the process of looking for prostate cancer can be. Men usually go first to their family doctor for a digital rectal examination — but prostate cancer is difficult to pick up in its early stages using this technique. Elevated levels of a marker called prostate specific antigen can be a warning sign, but this test is not usually ordered for men below age 55. Ultrasound scans are often used to detect signs of cancer and guide biopsies, but the conventional low-resolution machines were designed to monitor fetuses or larger organs such as the heart. An MRI scan has better resolution but they are expensive to perform, so are often used only on patients deemed at higher risk.

“Small suspicious lesions that are too small to see on conventional ultrasound are frequently missed,” says Sandler.

Conventional ultrasound functions at 6-9 MHz; Exact Imaging’s technology functions at the much shorter wavelength of 29 MHz. That resolution can detect small, early-stage cancers and guide doctors to their exact location to take biopsy samples for analysis.

Sandler’s cancer had not metastasized and, in early September, he underwent surgery. “Miraculously, I was back at work in seven weeks,” he says. “There are so many terrible stories — Gord Downie is just one of them — where a serious cancer has no cure or a treatable cancer is detected too late. I was incredibly lucky,” says Sandler.

“To think that we found it, treated it without chemo and I’m back exercising four times a week and playing with my kids is simply unimaginable to me. Tech works. And I proved it.”

KalGene Pharmaceuticals closes financing to support the clinical development of its Alzheimer’s therapeutic

November 27, 2017 / Portfolio News

KalGene Pharmaceuticals Inc. today announced the closing of a Series A financing to support the advancement of its lead Alzheimer’s therapeutic program.  The syndicated investment was led by Lumira Capital and included participation from Anges Québec, Anges Québec Capital, Accel-Rx Health Sciences Accelerator and a number of Canadian family offices.  In addition to the equity financing the company has also received significant financial support from leading Foundations focused on neurodegenerative diseases.  Proceeds from these combined funding sources will support the development of KalGene’s lead Alzheimer’s therapeutic candidate.

“We’ve followed the development of this program for many years and have been working directly with the team for the last year on this opportunity. During the past year, one of Lumira’s advisors, Dr. John Gillard, joined the company as Vice President Drug Development to advance the lead product to the clinic,” said Daniel Hetu, Managing Director at Lumira Capital.

“The company has made terrific progress as a result of its work with a number of key collaboration partners.  These partners include; the National Research Council of Canada, McGill University, where a number of the lead clinicians and researchers are located, and a number of agencies and foundations that continue to provide non-dilutive capital and innovation support including the Weston Brain Institute, Consortium Québécois sur la découverte du médicament (CQDM), Brain Canada and Ontario Brain Institute,” noted Jacki Jenuth, Partner at Lumira Capital.

“Anges Québec members and Anges Québec Capital were impressed by the quality of the science behind KalGene and their approach to Alzheimer’s treatments. We know that important challenges lie ahead in this race to tackle Alzheimer’s disease, but we are confident that the KalGene team has what it takes to succeed”, said François Gilbert, CEO of Anges Québec and Anges Québec Capital.

“One of the biggest obstacles to treating Alzheimer’s and other neurodegenerative diseases is delivering therapeutics past the blood-brain barrier,” said Natalie Dakers, President and CEO of Accel-Rx.  “We are pleased to be part of the team enabling KalGene to advance the development of its innovative solution to this problem, buoying hopes for an effective new treatment for this devastating disease.”

“We are very pleased to close on this financing which will allow us to complete our preclinical studies and move our program into the clinic and eventually have an impact on patients’ lives.”  said Dr. T. Nathan Yoganathan, President and Chief Scientific Officer of KalGene.

About KalGene Pharmaceuticals, Inc.

KalGene Pharmaceuticals Inc. is a pre-clinical stage company focussed on the development of precision medicine therapeutics to slow the progression of Alzheimer’s disease. Kalgene Pharmaceutical’s lead, KAL-ABP, targeting toxic amyloid beta oligomers, has been shown in animal models of Alzheimer’s Disease to cross the blood-brain barrier and to significantly reduce the plaque burden, leading to improved cognition and neuronal connectivity.

St. Joe’s receives research funding from Lumira Capital

November 22, 2017 / Lumira News

The Research Institute of St. Joe’s Hamilton is set to receive philanthropic funding from Lumira Capital to support research initiatives at the hospital.

Lumira Capital – a leading North American healthcare venture capital firm – has launched a new philanthropic initiative, the first of its kind in Canada. Up to fifteen healthcare foundations will benefit from a donated portion of Lumira Capital’s investment profits.

St. Joseph’s Healthcare Foundation has been selected as one of the beneficiaries of this award, along with other leading healthcare providers including the Princess Margaret Cancer Foundation & St. Michael’s Hospital.

As the funding is unrestricted, it allows the Research Institute the freedom to allocate the funds in a way that maximizes their impact on the research community of St. Joe’s.

“Being selected among some of the top health research institutions in Canada demonstrates the growing impact that we’ve been able to have,” says Dori Cartwright, Executive Director of the Research Institute of St. Joe’s Hamilton. “This funding will help us be able to grow the research enterprise at St. Joe’s – enabling us to have a greater impact on patients’ quality of life.”

Corvia Medical Announces Enrollment Of First Patients In Pivotal Trial Of The IASD® System II For The Treatment Of Heart Failure

November 14, 2017 / Portfolio News

ANAHEIM, CA – November 14, 2017 — Corvia Medical, Inc., a privately-held medical device company that developed a first-in-class transcatheter structural heart device to treat heart failure, today announced that it has enrolled the first patients in its global multicenter trial (REDUCE LAP-HF II) of the Interatrial Shunt Device (IASD) for the treatment of heart failure. Results from the randomized feasibility trial (REDUCE LAP-HF I) will be presented at the American Heart Association meeting on Wednesday, November 15th in Anaheim, CA.

Dr. Sanjiv Shah from Northwestern Memorial Hospital’s Center for Heart Failure at the Bluhm Cardiovascular Institute in Chicago will be presenting on behalf of the investigators, at the Innovative Therapies and Novel Applications session of the Late-Breaking Science program. The presentation, titled Transcatheter Interatrial Shunt Device for the Treatment of Heart Failure: Results from the REDUCE LAP-HF I Randomized Controlled Trial, will be presented at 9:00am PT on Wednesday, November 15th.

Dr. Rami Kahwash, heart failure cardiologist at the Ohio State University Wexner Medical Center enrolled the first patient in the REDUCE LAP-HF II trial, the next study to evaluate the IASD in support of US FDA approval. He commented, “The treatment of heart failure with preserved ejection fraction is challenging, and caring for these patients can be frustrating. Multiple randomized drug trials have demonstrated that conventional heart failure medications are ineffective in this type of heart failure and as a result, patients have limited therapeutic options.” Dr. Kahwash continues, “Since heart failure symptoms are directly linked to elevated left atrial pressures, the interatrial shunt device has the potential to offer an effective treatment by facilitating continuous and dynamic decompression of the left atrium, subsequently reducing symptoms and improving quality of life for this patient population.”

 

The REDUCE LAP-HF II trial is a prospective, multicenter, double blind randomized controlled trial to evaluate the IASD System to reduce elevated left atrial pressure in patients with heart failure with preserved or mid-range ejection fraction. The study will enroll patients at up to 70 sites in the U.S. and up to 30 sites outside the U.S. Dr. Sanjiv Shah and Dr. Ted Feldman from the NorthShore University HealthSystem in Evanston, Illinois, serve as co-principal investigators for the study.

 

“Having experience with the IASD in prior studies, I have substantial appreciation for the elegance of the implant procedure and have been very satisfied with the positive effect it has had,” commented Dr. Ted Feldman, “The open label study showed that following IASD implantation, patients had significantly fewer heart failure symptoms and could exercise significantly longer, giving them a substantially better quality of life. We are pleased to be part of the pivotal study and continue to offer this innovative treatment to our heart failure patients.”

“Enrolling the first patients in the pivotal trial is a major step toward bringing this innovative technology to patients in the United States,” said George Fazio, President and CEO of Corvia Medical. “This large randomized trial demonstrates our commitment to building a body of strong clinical evidence to meet a significant unmet clinical need by advancing care for patients suffering with the debilitating symptoms of heart failure.”

About the InterAtrial Shunt Device (IASD®)

The IASD is the world’s first transcatheter device approved in the European Union to treat heart failure with preserved or mid-range ejection fraction (HFpEF/HFmrEF). After creating a small opening in the atrial septum, the IASD implant is deployed, forming a passage between the left and right atria that enables the left atrium to decompress at rest and during physical activity, with the aim of lowering left atrial pressure. By facilitating continuous and dynamic decompression of the left atrium, the IASD aims to reduce heart failure symptoms, improve quality of life, decrease heart failure hospitalization rates, and reduce the overall cost burden of managing heart failure patients. For more information, please visit http://treatmyheartfailure.com/. The IASD is an investigational device and not available for commercial distribution in the United States.

About Corvia Medical, Inc.

Corvia Medical, Inc., formerly DC Devices Inc., is dedicated to revolutionizing the treatment of heart failure with first-in-class transcatheter structural heart devices. Privately held, the company is backed by Third Rock Ventures, General Catalyst Partners, AccelMed, Lumira Capital and an undisclosed strategic investor. For more information, please visit http://corviamedical.com/

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